From organs to muscle tissue: how stem cells are being used in 3D

A Sunday Afternoon on the Island of La Grande Jatte by Georges-Pierre Seurat

When most people think of stem cells, they might conjure up an image of small dots under a microscope. It is hard to imagine these small specs being applied to three-dimensional structures. But like a pointillism painting, such as A Sunday Afternoon on the Island of La Grande Jatte by Georges-Pierre Seurat, stem cells can be used to help build things never thought possible. Two studies demonstrate this concept in very different ways.

MIT engineers have designed coiled “nanoyarn,” shown as an artist’s interpretation here. The twisted fibers are lined with living cells and may be used to repair injured muscles and tendons while maintaining their flexibility. Image by Felice Frankel

A study at MIT used nanofiber coated with muscle stem cells and mesenchymal stem cells in an effort to provide a flexible range of motion for these stem cells. Hundreds of thousands of nanofibers were twisted, resembling yarn and rope, in order to mimic the pattern found in tendons and muscle tissue throughout the body. The researchers at MIT found that the yarn like structure of the nanofibers keep the stem cells alive and growing, even as the team stretched and bent the fibers multiple times.

Normally, when a person injures these types of tissues, particularly around a major joint such as the shoulder or knee, it require surgery and weeks of limited mobility to heal properly. The MIT team hopes that their technology could be applied toward treating the site of injury while maintaining range of motion as the newly applied stem cells continue to grow to replace the injured tissue.

In an article, Dr. Ming Guo, assistant professor of mechanical engineering at MIT and one of the authors of the study, was quoted as saying,

“When you repair muscle or tendon, you really have to fix their movement for a period of time, by wearing a boot, for example. With this nanofiber yarn, the hope is, you won’t have to wearing anything like that.”

Their complete findings were published in the Proceedings of the National Academy of Sciences (PNAS).

Researchers in Germany have created transparent human organs using a new technology that could pave the way to print three-dimensional body parts such as kidneys for transplants. Above, Dr. Ali Ertuerk inspects a transparent human brain.
Photo courtesy of Reuters.

In a separate study, researchers in Germany have successfully created transparent human organs, paving the way to print three-dimensional body parts. Dr. Ali Erturk at Ludwig Maximilians University in Munich, with a team of scientists, developed a technique to create a detailed blueprint of organs, including blood vessels and every single cell in its specific location. These directions were then used to print a scaffold of the organ. With the help of a 3D printer, stem cells, acting like ink in a printer, were injected into the correct positions to make the organ functional.

Previously, 3D-printed organs lacked detailed cellular structures because they were based on crude images from computer tomography or MRI machines. This technology has now changed that.

In an article, Dr. Erturk is quoted as saying,

“We can see where every single cell is located in transparent human organs. And then we can actually replicate exactly the same, using 3D bioprinting technology to make a real functional organ. Therefore, I believe we are much closer to a real human organ for the first time now.”

Stem Cell Agency Awards Almost $4 Million to Develop a Treatment for Spinal Degeneration

Today the governing Board of the California Institute for Regenerative Medicine (CIRM) awarded $3.9 million to Ankasa Regenerative Therapeutics for a promising approach to treat a degenerative condition that can cause chronic, progressive back pain.

As we get older, the bones, joints and ligaments in our back become weak and less able to hold the spinal column in alignment.  As a result, an individual vertebral bone in our spine may slip forward over the one below it, compressing the nerves in the spine, and causing lower back pain or radiating pain.  This condition, called degenerative spondylolisthesis, primarily affects individuals over the age of 50 and, if left untreated, can cause intense pain and further degeneration of adjacent regions of the spine.

Current treatment usually involves taking bone from one of the patient’s other bones, and moving it to the site of the injury.  The transplanted bone contains stem cells necessary to generate new bone.  However, there is a caveat to this approach— as we get older the grafts become less effective because the stem cells in our bones are less efficient at making new bone.  The end result is little or no bone healing. 

Ankasa has developed ART352-L, a protein-based drug product meant to enhance the bone healing properties of these bone grafts.  ART352-L works by stimulating bone stem cells to  increase the amount of bone produced by the graft.

The award is in the form of a CLIN1 grant, with the goal of completing the testing needed to apply to the Food and Drug Administration (FDA) for permission to start a clinical trial in people.

This is a project that CIRM has supported through earlier phases of research.

“We are excited to see the development that this approach has made since its early stages and reflects our commitment to supporting the most promising science and helping it advance to the clinic,” says Maria T. Millan, MD, President & CEO of CIRM. “There is an unmet medical need in older patients with bone disorders such as degenerative spondylolisthesis.  As our population ages, it is important for us to invest in potential treatments such as these that can help alleviate a debilitating condition that predisposes to additional and fatal medical complications.”

See the animated video below for a descriptive and visual synopsis of degenerative spondylolisthesis.

Stem cell byproducts provide insight into cure for spina bifida

A diagram of an infant born with spina bifida, a birth defect where there is an incomplete closing of the backbone portion of the spinal cord. Photo courtesy of the Texas Children’s Hospital website.

Some of you might remember a movie in the early 2000s by the name of “Miracle in Lane 2”. The film is based on an inspirational true story and revolves around a boy named Justin Yoder entering a soapbox derby competition. In the movie, Justin achieves success as a soapbox derby driver while adapting to the challenges of being in a wheelchair.

Scene from “Miracle in Lane 2”

The reason that Justin is unable to walk is due to a birth defect known as spina bifida, which causes an incomplete closing of the backbone portion of the spinal cord, exposing tissue and nerves. In addition to difficulties with walking, other problems associated with this condition are problems with bladder or bowel control and accumulation of fluid in the brain.

According to the Center for Disease Control (CDC) , each year about 1,645 babies in the US are born with spina bifida, with Hispanic women having the highest rate of children born with the condition. There is currently no cure for this condition, but researchers at UC Davis are one step closer to changing that.

Dr. Aijun Wang examining cells under a microscope. He has identified stem cell byproducts that protect neurons. Photo courtesy of UC Regents/UC Davis Health

Dr. Aijun Wang, Dr. Diana Farmer, and their research team have identified crucial byproducts produced by stem cells that play an important role in protecting neurons. These byproducts could assist with improving lower-limb motion in patients with spina bifida.

Prior to this discovery, Dr. Farmer and Dr. Wang demonstrated that prenatal surgery combined with connective tissue (e.g. stromal cells) derived from stem cells improved hind limb control in dogs with spina bifida. Below you can see a clip of two English bulldogs with spina bifida who are now able to walk.

Their findings were published in the Journal of the Federation of American Societies for Experimental Biology on February 12, 2019.

The team will use their findings to perfect the neuroprotective qualities of a stem cell treatment developed to improve locomotive problems associated with spina bifida.

In a public release posted by EurekaAlert!, Dr. Wang is quoted as saying, “We are excited about what we see so far and are anxious to further explore the clinical applications of this research.”

The discovery and development of a treatment for spina bifida was funded by a $5.66 million grant from CIRM. You can read more about that award and spina bifida on a previous blog post linked here.

Gene therapy gives patient a cure and a new lease on life

Brenden Whittaker (left), of Ohio, is a patient born with a rare genetic immune disease who was treated at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center in a CIRM funded gene therapy trial. Dr. David Williams (on right) is Brenden’s treating physician.
Photo courtesy of Rose Lincoln – Harvard Staff Photographer

Pursuing an education can be quite the challenge in itself without the added pressure of external factors. For Brenden Whittaker, a 25 year old from Ohio, the constant trips to the hospital and debilitating nature of an inherited genetic disease made this goal particularly challenging and, for most of his life, out of sight.

Brenden was born with chronic granulomatous disease (CGD), a rare genetic disorder that affects the proper function of neutrophils, a type of white blood cell that is an essential part of the body’s immune system. This leads to recurring bacterial and fungal infections and the formation of granulomas, which are clumps of infected tissue that arise as the body attempts to isolate infections it cannot combat. People with CGD are often hospitalized routinely and the granulomas themselves can obstruct digestive pathways and other pathways in the body. Antibiotics are used in an attempt to prevent infections from occurring, but eventually patients stop responding to them. One in two people with CGD do not live past the age of 40.

In Brenden’s case, when the antibiotics he relied on started failing, the doctors had to resort to surgery to cut out an infected lobe of his liver and half his right lung. Although the surgery was successful, it would only be a matter of time before a vital organ was infected and surgery would no longer be an option.

This ultimately lead to Brenden becoming the first patient in a CGD gene therapy trial at the Dana-Farber/Boston Children’s Cancer and Blood Disorders Center.  The trial, lead by UCLA’s Dr. Don Kohn thanks to a CIRM grant, combats the disease by correcting the dysfunctional gene inside a patient’s blood stem cells. The patient’s corrected blood stem cells are then reintroduced, allowing the body to produce properly functioning neutrophils, rebooting the immune system.

It’s been a little over three years since Brenden received this treatment in late 2015, and the results have been remarkable. Dr. David Williams, Brenden’s treating physician, expected Brenden’s body to produce at least 10 percent of the functional neutrophils, enough so that Brenden’s immune system would provide protection similar to somebody without CGD. The results were over 50 percent, greatly exceeding expectations.

Brenden Whittaker mowing the lawn in the backyard of his home in Columbus, Ohio. He is able to do many more things without the fear of infection since participating in the trial. Photo courtesy of Colin McGuire

In an article published by The Harvard Gazette, Becky Whittaker, Brendan’s mother, is quoted as saying, ““Each day that he’s free of infection, he’s able to go to class, he’s able to work at his part-time job, he’s able to mess around playing with the dog or hanging out with friends…[this] is a day I truly don’t believe he would have had beyond 2015 had something not been done.”

In addition to the changes to his immune system, the gene therapy has reinvigorated Brenden’s drive for the future. Living with CGD had caused Brenden to miss out on much of his schooling throughout the years, having to take constant pauses from his academics at a community college. Now, Brenden aims to graduate with an associate’s degree in health sciences in the spring and transfer to Ohio State in the fall for a bachelor’s degree program. In addition to this, Brenden now has dreams of attending medical school.

In The Harvard Gazette article, Brenden elaborates on why he wants to go to medical school saying, ” Just being the patient for so long, I want to give back. There are so many people who’ve been there for me — doctors, nurses who’ve been there for me [and] helped me for so long.”

In a press release dated February 25, 2019, Orchard Therapeutics, a biopharmaceutical company that is continuing the aforementioned approach for CGD, announced that six patients treated have shown adequate neutrophil function 12 months post treatment. Furthermore, these six patients no longer receive antibiotics related to CGD. Orchard Therapeutics also announced that they are in the process of designing a registrational trial for CGD.

Gene therapy and blood stem cells cure sickle cell disease patients

Sickle-shaped blood cells. The cells become lodged in blood vessels, causing strokes or excruciating pain as blood stops flowing. Photo courtesy of Omikron/Science Source

Blood is the lifeline of the body. The continuous, unimpeded circulation of blood maintains oxygen flow throughout the body and enables us to carry out our everyday activities. Unfortunately, there are individuals whose own bodies are in a constant battle that prevents this from occurring seamlessly. They have something known as sickle cell disease (SCD), an inherited condition caused by a mutation in a single gene. Rather than producing normal, circular red blood cells, their bodies produce sickle shaped cells (hence the name) that can become lodged in blood vessels, preventing blood flow. The lack of blood flow can cause agonizing pain, known as crises, as well as strokes. Chronic crises can cause organ damage, which can eventually lead to organ failure. Additionally, since the misshapen cells don’t survive long in the body, people with SCD have a greater risk of being severely anemic and are more prone to infections. Monthly blood transfusions are often needed to help temporarily alleviate symptoms. Due to the debilitating nature of SCD, important aspects of everyday life such as employment and health insurance can be extremely challenging to find and maintain.

An estimated 100,000 people in the United States are living with SCD. Around the world, about 300,000 infants are born with the condition each year, a statistic that will increase to 400,000 by 2050 according to one study. Many people with SCD do not live past the age of 50. It is most prevalent in individuals with sub-Saharan African descent followed by people of Hispanic descent. Experts have stated that advances in treatment have been limited in part because SCD is concentrated in poorer minority communities.

Despite these grim statistics and prognosis, there is hope.

The New York Times and Boston Herald recently released featured articles that tell the personal stories of patients enrolled in a clinical trial conducted by bluebird bio. The trial uses gene therapy in combination with hematopoietic (blood) stem cells (HSCs) to give rise to normal red blood cells in SCD patients.

Here are the stories of these patients. To read the full New York Times article, click here. For the Boston Herald article, click here.

Brothers, Emmanuel “Manny” 21 and Aiden Johnson 7 at their home in Brockton, Massachusetts. Both brothers were born with sickle cell disease. Photo courtesy of Matt Stone for MediaNews Group/Boston Herald

Emmanuel “Manny” Johnson was the very first patient in the SCD trial. He was motivated to participate in the trial not just for himself but for his younger brother Aiden Johnson, who was also born with SCD. Manny has a tattoo with Aiden’s name written inside a red sickle cell awareness ribbon.

In the article Manny is quoted as saying “It’s not only that we share the same blood disease, it’s like I have to do better for him.”

Since receiving the treatment, Manny’s SCD symptoms have disappeared.

Brandon Williams received the stem cell gene therapy to replace sickle cells with healthy red blood cells. The tattoo on his right forearm is in honor of his sister, Britney, who died of sickle cell disease. Photo courtesy of Alyssa Schukar for The New York Times

For Brandon Williams of Chicago, the story of SCD is a very personal one. At just 21 years old, Brandon had suffered four strokes by the time he turned 18. His older sister, Britney Williams, died of sickle cell disease at the age of 22. Brandon was devastated and felt that his own life could end at any moment. He was then told about the SCD trial and decided to enroll. Following the treatment, his symptoms have vanished along with the pain and fear inflicted by the disease.

Carmen Duncan participated in the stem cell gene therapy trial and no longer has sickle-cell symptoms. She wants to join the military, something that wasn’t an option until now. Photo courtesy of Sean Rayford for The New York Times

The NY Times piece also profiles Carmen Duncan, a 20 year old from Charleston, South Carolina. She had her spleen removed when she was just two years old as a result of complications form SCD. Duncan spent a large portion of her childhood in hospitals, coping with the pain in her arms and legs from blocked blood vessels. She enrolled in the SCD trial as well and she no longer has any signs of SCD. Duncan had aspirations to join the military but was unable to because of her condition. Now that she is symptom free, she plans to enlist.

This SCD clinical trial has multiple trial sites across the US, one which is the UCSF Alpha Stem Cell Clinic , a CIRM funded clinic specializing in the delivery of stem cell clinical trials to patients. CIRM awarded a $7,999,999 grant to help establish this site.

New Study on Humans Shows Promise for Sepsis Therapy

A new study published in STEM CELLS, conducted by researchers at the University of Amsterdam, shows how mesenchymal stem cells (MSCs) can restore the health and improve the function of the immune system,  which could benefit the treatment of sepsis. Sepsis is a life-threatening complication from an infection that can lead to multiple organ failure. It is a major cause of illness and death worldwide and despite the use of antibiotics it kills about one in every four patients who contract it.

Since early studies done on animals have shown that treating sepsis with MSCs can reduce the mortality rate by as much as 73 percent, a group of researchers from University of Amsterdam sought to answer this question:  could humans realize the same benefits?

So, the team conducted an experiment by taking a group of healthy volunteers and inducing endotoxemia in them, where bacterial toxins can build up and cause fever, nausea and vomiting but do not cause long-term harm to the participants (?).  The idea was that by inducing endotoxemia, which exhibits some of the key characteristics of sepsis, that they could model the condition in people.

One hour prior to the initial dose, each person was given an infusion of either adipose (fat) mesenchymal stem cells (ACSs) taken from a donor,  or a placebo as a control. Those receiving the ASCs were divided into three groups, with each group receiving a consecutively higher dose of cells.

In a news release, Desiree Perlee, senior author of the study, said the study provided some valuable insights and information:

gI_100288_foto_perlee.png

Desirée Perlee

“The results showed that the ASCs were well tolerated…We realize that there is a limitation with the endotoxemia model. Although in a qualitative way it resembles responses seen in patients with sepsis, it differs in that sepsis-associated alterations are more severe and sustained, while in the endotoxemia model responses occur in a very rapid, short-lived and transient way. But despite these limitations, some of our findings confirm the earlier studies on animals. We believe they show further testing of ASCs in actual sepsis patients is warranted.”

Dr. Jan Nolta, Editor-in-Chief of STEM CELLS (and a CIRM-grantee), said, “This novel clinical trial provides important insight into the mechanism of action of MSCs in inflammation and provides human safety data in support of treatment of sepsis using MSCs.

 

Starving stem cells of oxygen can help build stronger bones

Leach_Kent_BME.2012

J. Kent Leach: Photo courtesy UC Davis

We usually think that starving something of oxygen is going to make it weaker and maybe even kill it. But a new study by J. Kent Leach at UC Davis shows that instead of weakening bone defects, depriving them of oxygen might help boost their ability to create new bone or repair existing bone.

Leach says in the past the use of stem cells to repair damaged or defective bone had limited success because the stem cells often didn’t engraft in the bone or survive long if they did. That was because the cells were being placed in an environment that lacked oxygen (concentration levels in bone range from 3% to 8%) so the cells found it hard to survive.

However, studies in the lab had shown that if you preconditioned mesenchymal stem cells (MSCs), by exposing them to low oxygen levels before you placed them on the injury site, you helped prolong their viability. That was further enhanced by forming the MSCs into three dimensional clumps called spheroids.

Lightbulb goes off

In the  current study, published in Stem Cells, Leach says the earlier spheroid results  gave him an idea:

“We hypothesized that preconditioning MSCs in hypoxic (low oxygen) culture before spheroid formation would increase cell viability, proangiogenic potential (ability to create new blood vessels), and resultant bone repair compared with that of individual MSCs.”

So, the researchers placed one group of human MSCs, taken from bone marrow, in a dish with just 1% oxygen, and another identical group of MSCs in a dish with normal oxygen levels. After three days both groups were formed into spheroids and placed in an alginate hydrogel, a biopolymer derived from brown seaweed that is often used to build cellular cultures.

Seaweed

Brown seaweed

The team found that the oxygen-starved cells lasted longer than the ones left in normal oxygen, and the longer those cells were deprived of oxygen the better they did.

Theory is great, how does it work in practice?

Next was to see how those two groups did in actually repairing bones in rats. Leach says the results were encouraging:

“Once again, the oxygen-deprived, spheroid-containing gels induced significantly more bone healing than did gels containing either preconditioned individual MSCs or acellular gels.”

The team say this shows the use of these oxygen-starved cells could be an effective approach to repairing hard-to-heal bone injuries in people.

“Short‐term exposure to low oxygen primes MSCs for survival and initiates angiogenesis (the development of new blood vessels). Furthermore, these pathways are sustained through cell‐cell signaling following spheroid formation. Hypoxic (low oxygen) preconditioning of MSCs, in synergy with transplantation of cells as spheroids, should be considered for cell‐based therapies to promote cell survival, angiogenesis, and bone formation.”

CIRM & Dr. Leach

While CIRM did not fund this study we have invested more than $1.8 million in another study Dr. Leach is doing to develop a new kind of imaging technology that will help us see more clearly what is happening in bone and cartilage-targeted therapies.

In addition, back in March of 2012, Dr. Leach spoke to the CIRM Board about his work developing new approaches to growing bone.

 

CIRM Supported Scientist Makes Surprising Discovery with Parasitic Gut Worms

 

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Image of gut lining and parasites.  Photo courtesy of UCSF/ Michael Fortes

 

It’s no secret that researchers have long believed adult stem cells could contribute to wound healing in the gut and skin, but in a new paper in Nature — a group of scientists at UC San Francisco made a surprising discovery.

Through several experiments using parasitic worms in the mouse gut, they found that as parasites dug into the intestinal walls of mice, the gut responded in an unexpected way – by reactivating a type of cell growth previously seen in fetal tissues.

So why is this important?

Simply put, it gives scientists new targets to go after. According to UCSF CIRM supported scientist Ophir Klein, MD, Ph.D., this discovery could be paradigm-shifting in terms of our understanding of how the mammalian body can repair damage and could help scientists develop more ways to enhance the body’s natural healing abilities.

Adult stem cells in the intestines are vital for maintaining the digestive status quo. The gut lining is made up of epithelial cells which absorb nutrients and produce protective mucus. These cells are replaced every few days by the stem cells at the base of crypts — indentations in the gut lining. Researchers expected that the same stem cells could also help repair tears in the gut.

How did they do it?

Larvae from parasites like H. polygyrus invade the gut lining in a mouse’s intestine, burying themselves to develop in the tissue. Based on prevailing ideas in the field, the scientists predicted that, in response, nearby stem cells would increase their productivity and patch up the worm-created wounds, but that is not what happened.

Instead, signs of the stem cells in worm-infected areas disappeared entirely; fluorescent markers that should have been expressed by one of the genes in the regular stem cell program completely vanished. And yet, even with no identifiable stem cells in the area, the wounded tissue regenerated more quickly than ever.

Researchers spent years trying to resolve this mystery and after a number of false starts and dead ends, the team eventually noticed the recurrence of a different gene, known as Sca-1.

Using antibody staining for the Sca-1 protein, the researchers realized that where the stem cell genes had disappeared, a different gene program was expressed instead: one that resembled the way that mouse guts develop in utero.

Upon their discovery, the researchers wondered whether the reactivation of this fetal program was a specific response to parasite infections, or if it could be a general strategy for many kinds of gut injury. Additional experiments showed that shutting down gut stem cells with irradiation or genetically targeting them for destruction triggered aspects of the same response: despite an absence of detectable stem cell activity, undifferentiated tissue grew rapidly nonetheless.

Later, once the acute injury is repaired, the gut may return to the normal stem cell program of producing differentiated cells that perform specific functions.

Many other injured tissues could benefit from the ability to quickly and efficiently make generalized repairs before returning to specialized adult cell production, opening up therapeutic opportunities. For example, developing treatments that bestow an ability to control the change between adult and fetal genetic programs might offer new strategies to manage conditions such as inflammatory bowel disease (IBD).

Stem Cell Roundup: New understanding of Huntington’s; how stem cells can double your DNA; and using “the Gary Oldman of cell types” to reverse aging

This week’s roundup highlights how we are constantly finding out new and exciting ways that stem cells could help change the way we treat disease.

Our Cool Stem Cell Image of the Week comes from our first story, about unlocking some of the secrets of Huntington’s disease. It comes from the Laboratory of Stem Cell Biology and Molecular Embryology at The Rockefeller University

Huntington's neurons

A new approach to studying and developing therapies for Huntington’s disease

Researchers at Rockefeller University report new findings that may upend the way scientists study and ultimately develop therapies for Huntington’s disease, a devastating, inherited neurodegenerative disorder that has no cure. Though mouse models of the disease are well-established, the team wanted to focus on human biology since our brains are more complex than those of mice. So, they used CRISPR gene editing technology in human embryonic stem cells to introduce the genetic mutations that cause HD.

Though symptoms typically do not appear until adulthood, the researchers were surprised to find that in their human cell-based model of HD, abnormalities in nerve cells occur at the earliest steps in brain development. These results suggest that HD therapies should focus on treatments much earlier in life.

The researchers observed another unexpected twist: cells that lack Huntingtin, the gene responsible for HD, are very similar to cells found in HD. This suggests that too little Huntingtin may be causing the disease. Up until now, the prevailing idea has been that Huntington’s symptoms are caused by the toxicity of too much mutant Huntingtin activity.

We’ll certainly be keeping an eye on how further studies using this new model affect our understanding of and therapy development for HD.

This study was published in Development and was picked by Science Daily.

How you can double your DNA

dna

As you can imagine we get lots of questions about stem cell research here at CIRM. Last week we got an email asking if a stem cell transplant could alter your DNA? The answer is, under certain circumstances, yes it could.

A fascinating article in the Herald Review explains how this can happen. In a bone marrow transplant bad blood stem cells are killed and replaced with healthy ones from a donor. As those cells multiply, creating a new blood supply, they also carry the DNA for the donor.

But that’s not the only way that people may end up with dual DNA. And the really fascinating part of the article is how this can cause all sorts of legal and criminal problems.

One researcher’s efforts to reverse aging

gary-oldman

Gary Oldman: Photo courtesy Variety

“Stem cells are the Gary Oldman of cell types.” As a fan of Gary Oldman (terrific as Winston Churchill in the movie “Darkest Hour”) that one line made me want to read on in a profile of Stanford University researcher Vittorio Sebastiano.

Sebastiano’s goal is, to say the least, rather ambitious. He wants to reverse aging in people. He believes that if you can induce a person’s stem cells to revert to a younger state, without changing their function, you can effectively turn back the clock.

Sebastiano says if you want to achieve big things you have to think big:

“Yes, the ambition is huge, the potential applications could be dramatic, but that doesn’t mean that we are going to become immortal in some problematic way. After all, one way or the other, we have to die. We will just understand aging in a better way, and develop better drugs, and keep people happier and healthier for a few more years.”

The profile is in the journal Nautilus.

Family, faith and funding from CIRM inspire one patient to plan for his future

Caleb Sizemore speaks to the CIRM Board at the June 2017 ICOC meeting.

Having been to many conferences and meetings over the years I have found there is a really simple way to gauge if someone is a good speaker, if they have the attention of people in the room. You just look around and see how many people are on their phones or laptops, checking their email or the latest sports scores.

By that standard Caleb Sizemore is a spellbinding speaker.

Last month Caleb spoke to the CIRM Board about his experiences in a CIRM-funded clinical trial for Duchenne Muscular Dystrophy. As he talked no one in the room was on their phone. Laptops were closed. All eyes and ears were on him.

To say his talk was both deeply moving and inspiring is an understatement. I could go into more detail but it’s so much more powerful to hear it from  Caleb himself. His words are a reminder to everyone at CIRM why we do this work, and why we have to continue to do all that we can to live up to our mission statement and accelerate stem cell treatments to patients with unmet medical needs.

Video produced by Todd Dubnicoff/CIRM


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